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An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)

NCT05025332 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2025-06-18

Study results available
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Summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Pitt Hopkins Syndrome.

Conditions

  • Pitt Hopkins Syndrome

Interventions

DRUG

NNZ-2591

NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.

Sponsors & Collaborators

  • Neuren Pharmaceuticals Limited

    lead INDUSTRY

Principal Investigators

  • James Shaw · Neuren Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-14
Primary Completion
2024-02-27
Completion
2024-05-03
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05025332 on ClinicalTrials.gov